BR112021008832A2 - Proteínas de núcleo de mininucleossoma e uso em distribuição de ácido nucleico - Google Patents

Proteínas de núcleo de mininucleossoma e uso em distribuição de ácido nucleico Download PDF

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Publication number
BR112021008832A2
BR112021008832A2 BR112021008832-6A BR112021008832A BR112021008832A2 BR 112021008832 A2 BR112021008832 A2 BR 112021008832A2 BR 112021008832 A BR112021008832 A BR 112021008832A BR 112021008832 A2 BR112021008832 A2 BR 112021008832A2
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BR
Brazil
Prior art keywords
mininucleosome
domain
nucleic acid
modified polypeptide
amino acid
Prior art date
Application number
BR112021008832-6A
Other languages
English (en)
Portuguese (pt)
Inventor
Adarsha Koirala
Original Assignee
Summation Bio, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Summation Bio, Inc. filed Critical Summation Bio, Inc.
Publication of BR112021008832A2 publication Critical patent/BR112021008832A2/pt

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/35—Fusion polypeptide containing a fusion for enhanced stability/folding during expression, e.g. fusions with chaperones or thioredoxin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Inorganic Chemistry (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
BR112021008832-6A 2018-11-08 2019-11-06 Proteínas de núcleo de mininucleossoma e uso em distribuição de ácido nucleico BR112021008832A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862757683P 2018-11-08 2018-11-08
US62/757,683 2018-11-08
PCT/US2019/060119 WO2020097235A1 (en) 2018-11-08 2019-11-06 Mini-nucleosome core proteins and use in nucleic acid delivery

Publications (1)

Publication Number Publication Date
BR112021008832A2 true BR112021008832A2 (pt) 2021-08-31

Family

ID=70611091

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021008832-6A BR112021008832A2 (pt) 2018-11-08 2019-11-06 Proteínas de núcleo de mininucleossoma e uso em distribuição de ácido nucleico

Country Status (12)

Country Link
US (1) US20210395303A1 (ja)
EP (1) EP3877526A4 (ja)
JP (2) JP2022512977A (ja)
KR (1) KR20210108952A (ja)
CN (1) CN113423834A (ja)
AU (1) AU2019376641A1 (ja)
BR (1) BR112021008832A2 (ja)
CA (1) CA3118146A1 (ja)
IL (1) IL282982A (ja)
MX (1) MX2021005313A (ja)
SG (1) SG11202104354TA (ja)
WO (1) WO2020097235A1 (ja)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102470059B1 (ko) * 2018-05-18 2022-11-22 에프. 호프만-라 로슈 아게 큰 핵산의 표적화된 세포내 전달
IL297250A (en) * 2020-04-13 2022-12-01 Summation Bio Inc Modified mininucleosome core proteins and use in nucleic acid delivery
CA3226457A1 (en) * 2021-07-30 2023-02-02 Amy WALKER Nanoparticles and peptides for the delivery of cargos to muscle cells
EP4124345A1 (en) * 2021-07-30 2023-02-01 4basebio UK Ltd Nanoparticles for muscle delivery
WO2023014724A2 (en) * 2021-08-03 2023-02-09 Summation Bio, Inc. Scaffold matrix attachment regions for gene therapy

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5670347A (en) * 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
WO1996040958A1 (en) * 1995-06-07 1996-12-19 Baylor College Of Medicine Nucleic acid transporters for delivery of nucleic acids into a cell
WO1996041606A2 (en) * 1995-06-08 1996-12-27 Therexsys Limited Improved pharmaceutical compositions for gene therapy
AU1029099A (en) * 1997-10-10 1999-05-03 Aventis Pharma Deutschland Gmbh Transfection system for the transfer of nucleic acids into cells
KR20040011480A (ko) * 2001-03-22 2004-02-05 덴드레온 샌 디에고 엘엘씨 세린 프로테아제 ｃｖｓｐ14를 암호화하는 핵산 분자,암호화된 폴리펩티드 및 이에 근거한 방법
DK1594973T3 (da) * 2003-02-10 2012-02-13 Max Delbrueck Centrum Transposonbaseret målretningssystem
JP4521519B2 (ja) * 2003-05-26 2010-08-11 学校法人慶應義塾 部分ヒストンテイル型イムノポーター
CA2528830A1 (en) * 2003-06-10 2004-12-16 Toolgen, Inc. Transducible dna-binding proteins
US7205387B2 (en) * 2003-08-28 2007-04-17 Agency For Science, Technology And Research Recombinant polypeptide useful for neurotrophin receptor mediated gene delivery and as neurotrophin agonist
US20060018911A1 (en) * 2004-01-12 2006-01-26 Dana Ault-Riche Design of therapeutics and therapeutics
PL391627A1 (pl) * 2010-06-25 2012-01-02 Adamed Spółka Z Ograniczoną Odpowiedzialnością Przeciwnowotworowe białko fuzyjne
US9746475B2 (en) * 2011-03-14 2017-08-29 University Of Southern California Antibody and antibody mimetic for visualization and ablation of endogenous proteins
US10407469B2 (en) * 2016-04-01 2019-09-10 Northwestern University Programmable polypeptide and nucleic acid nanoparticles
AU2017378427A1 (en) * 2016-12-14 2019-06-20 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery

Also Published As

Publication number Publication date
SG11202104354TA (en) 2021-05-28
JP2022512977A (ja) 2022-02-07
WO2020097235A1 (en) 2020-05-14
CA3118146A1 (en) 2020-05-14
MX2021005313A (es) 2021-10-13
KR20210108952A (ko) 2021-09-03
IL282982A (en) 2021-06-30
CN113423834A (zh) 2021-09-21
US20210395303A1 (en) 2021-12-23
EP3877526A1 (en) 2021-09-15
AU2019376641A1 (en) 2021-05-27
JP2024029097A (ja) 2024-03-05
EP3877526A4 (en) 2023-03-29

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